US20240018492A1 - Prime editing using hiv reverse transcriptase and cas9 or variant thereof - Google Patents

Prime editing using hiv reverse transcriptase and cas9 or variant thereof Download PDF

Info

Publication number
US20240018492A1
US20240018492A1 US18/247,108 US202118247108A US2024018492A1 US 20240018492 A1 US20240018492 A1 US 20240018492A1 US 202118247108 A US202118247108 A US 202118247108A US 2024018492 A1 US2024018492 A1 US 2024018492A1
Authority
US
United States
Prior art keywords
variant
hiv
prime
reverse transcriptase
target
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
US18/247,108
Other languages
English (en)
Inventor
Jin-soo Kim
Chul-Yong Park
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Institute for Basic Science
Original Assignee
Institute for Basic Science
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Institute for Basic Science filed Critical Institute for Basic Science
Assigned to INSTITUTE FOR BASIC SCIENCE reassignment INSTITUTE FOR BASIC SCIENCE ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: KIM, JIN-SOO, PARK, CHUL-YONG
Publication of US20240018492A1 publication Critical patent/US20240018492A1/en
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241Nucleotidyltransferases (2.7.7)
    • C12N9/1276RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07Nucleotidyltransferases (2.7.7)
    • C12Y207/07049RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
US18/247,108 2020-09-29 2021-09-29 Prime editing using hiv reverse transcriptase and cas9 or variant thereof Pending US20240018492A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
KR20200126709 2020-09-29
KR10-2020-0126709 2020-09-29
PCT/KR2021/013326 WO2022071745A1 (fr) 2020-09-29 2021-09-29 Édition primaire utilisant la transcriptase inverse du vih et cas9 ou un variant de celui-ci

Publications (1)

Publication Number Publication Date
US20240018492A1 true US20240018492A1 (en) 2024-01-18

Family

ID=80951772

Family Applications (1)

Application Number Title Priority Date Filing Date
US18/247,108 Pending US20240018492A1 (en) 2020-09-29 2021-09-29 Prime editing using hiv reverse transcriptase and cas9 or variant thereof

Country Status (3)

Country Link
US (1) US20240018492A1 (fr)
KR (1) KR20230075420A (fr)
WO (1) WO2022071745A1 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024026415A1 (fr) * 2022-07-27 2024-02-01 The Trustees Of Columbia University In The City Of New York Compositions, systèmes et procédés de réécriture par matrice d'arn

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10308955B2 (en) * 2011-10-26 2019-06-04 Newvectys Transient expression vectors, preparation and uses thereof
BR102019009665A2 (pt) * 2018-12-21 2022-02-08 Jacques P. Tremblay Modificação da proteína precursora beta amiloide (app) através da edição de base usando o sistema crispr/cas9
JP2022526908A (ja) * 2019-03-19 2022-05-27 ザ ブロード インスティテュート,インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
CN111378051B (zh) * 2020-03-25 2022-03-01 北京市农林科学院 Pe-p2引导编辑系统及其在基因组碱基编辑中的应用

Also Published As

Publication number Publication date
KR20230075420A (ko) 2023-05-31
WO2022071745A1 (fr) 2022-04-07

Similar Documents

Publication Publication Date Title
Givens et al. Nanoparticle-based delivery of CRISPR/Cas9 genome-editing therapeutics
JP7245651B2 (ja) Crispr/cas構成成分のための脂質ナノ粒子製剤
JP6712948B2 (ja) 組成物、及びヌクレオチドリピート障害におけるcrispr−cas系の使用方法
CA3026112A1 (fr) Complexes cpf1 a activite d'indel reduite
Christie et al. Minireview: delivering the code: polyplex carriers for deoxyribonucleic acid and ribonucleic acid interference therapies
WO2019126589A1 (fr) Micelles pour la complexation et l'administration de protéines et d'acides nucléiques
EP3449004A1 (fr) Micelles à base de poly (histidine) pour la complexation et l'administration de protéines et d'acides nucléiques
CA3077251A1 (fr) Compositions et methodes pour l'edition du gene ttr et le traitement de l'amyloidose attr
JP2008520209A (ja) siRNAの有効なキャリアとしての高度に枝分かれしたHKペプチド
US20210403922A1 (en) Non-toxic cas9 enzyme and application thereof
Louzao et al. Hydrazone-modulated peptides for efficient gene transfection
US20220325264A1 (en) Modified nucleic acid editing systems for tethering donor dna
US20240018492A1 (en) Prime editing using hiv reverse transcriptase and cas9 or variant thereof
CN113194998A (zh) 用于非病毒递送用于基因编辑和视网膜基因治疗的质粒DNA的聚(β-氨基酯)纳米颗粒
CN113874076A (zh) 包括ttr向导rna和对rna引导的dna结合剂进行编码的多核苷酸的组合物和方法
Su et al. Cationic dynamic covalent polymers for gene transfection
CN113993994A (zh) 用于多肽表达的多核苷酸、组合物和方法
WO2023093862A1 (fr) Procédé de modulation de pcsk9 et ses utilisations
Hu et al. Nanobiomaterial vectors for improving gene editing and gene therapy
US20230151343A1 (en) Genome editing using cas9 or cas9 variant
WO2021215827A1 (fr) Édition du génome à l'aide de cas9 ou d'un variant cas9
EP4119166A1 (fr) Composition pour induire l'apoptose de cellules ayant une variation de séquence génomique et procédé pour induire l'apoptose de cellules à l'aide de la composition
Garcia et al. Peptide shuttle-mediated delivery for brain gene therapies
KR20240041835A (ko) 식물세포 소기관 dna의 염기 교정
KR20230139064A (ko) 식물세포 소기관 dna의 아데닌을 구아닌으로 염기 교정

Legal Events

Date Code Title Description
AS Assignment

Owner name: INSTITUTE FOR BASIC SCIENCE, KOREA, REPUBLIC OF

Free format text: ASSIGNMENT OF ASSIGNORS INTEREST;ASSIGNORS:KIM, JIN-SOO;PARK, CHUL-YONG;SIGNING DATES FROM 20230327 TO 20230404;REEL/FRAME:063217/0236

STPP Information on status: patent application and granting procedure in general

Free format text: DOCKETED NEW CASE - READY FOR EXAMINATION